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Cirak S, Arechavala-Gomeza V, Guglieri M, et al. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: An open-label, phase 2, dose-escalation study. Lancet 2011;378(9791):595-605
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(2011)
Lancet
, vol.378
, Issue.9791
, pp. 595-605
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Cirak, S.1
Arechavala-Gomeza, V.2
Guglieri, M.3
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